High throughput drug screening (HTS) is a powerful tool that can be used to identify potential drug candidates for rare neurological diseases. These diseases often have limited treatment options, and the development of new therapies can be challenging due to the small patient population and the complexity of the underlying disease mechanisms.

HTS involves the rapid screening of large libraries of compounds for their ability to interact with disease-specific targets or pathways. The screening process can be automated and scaled up to test hundreds of thousands or even millions of compounds in a short period of time.
To perform HTS for rare neurological diseases, researchers can use a variety of platforms, such as cell-based assays or biochemical assays, depending on the specific disease mechanism being targeted. For example, for diseases caused by mutations in ion channels, researchers may use electrophysiological assays to test the ability of compounds to modulate ion channel activity.
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One advantage of HTS is that it can identify new drug candidates for rare neurological diseases that may not have been previously considered. However, it is important to validate any hits identified in HTS with additional experiments to ensure that they are safe and effective for use in humans.
Overall, HTS can be a valuable tool for identifying new therapeutic options for rare neurological diseases, and it has the potential to accelerate the drug discovery process for these challenging conditions.

The video below is with Dr. Chris Austin with the National Institutes of Health (NIH) showing their drug screening process.